DGAP-News

RedHill Biopharma Announces YELIVA(R) (ABC294640) Abstract Presentation at the 2017 Cholangiocarcinoma Foundation Annual Conference - Seite 2

(NCI).

The authors of the abstract assessed the effects of YELIVA(R) on
cholangiocarcinoma cells in culture and in patients in the Phase I clinical
study with YELIVA(R), concluding that the findings from these studies
suggest that YELIVA(R) may be an effective drug for the treatment of
cholangiocarcinoma. Cholangiocarcinoma (bile duct cancer) is a highly
lethal malignancy for which there is a need for more effective systemic
treatments. Surgery with complete resection remains the only curative
therapy for cholangiocarcinoma, however only a minority of patients are
classified as having a resectable tumor at the time of diagnosis[2].

RedHill announced in June 2016 that the final results from the Phase I
study with YELIVA(R) in patients with advanced solid tumors confirmed that
the study, conducted at MUSC Hollings Cancer Center, successfully met its
primary and secondary endpoints, demonstrating that the drug is well-
tolerated and can be safely administered to cancer patients at doses that
provide circulating drug levels that are predicted to have therapeutic
activity.

Of the three patients with cholangiocarcinoma in the Phase I study, one
subject achieved a sustained partial response (Overall Survival (OS) = 20.3
months) and the other two subjects had stable disease (OS = 17.6 and 16.3
months).

YELIVA(R) is a Phase II-stage, proprietary, first-in-class, orally-
administered sphingosine kinase-2 (SK2) selective inhibitor with anticancer
and anti-inflammatory activities, targeting multiple oncology, inflammatory
and gastrointestinal indications. By inhibiting the SK2 enzyme, YELIVA(R)
blocks the synthesis of sphingosine 1-phosphate (S1P), a lipid signaling
molecule that promotes cancer growth and pathological inflammation.

A Phase II study with YELIVA(R) for the treatment of advanced
hepatocellular carcinoma (HCC) was initiated at MUSC Hollings Cancer
Center. The study is supported by a $1.8 million grant from the NCI,
awarded to MUSC, which is intended to fund a broad range of studies on the
feasibility of targeting sphingolipid metabolism for the treatment of a
variety of solid tumor cancers, with additional support from RedHill.

A Phase Ib/II study with YELIVA(R) for the treatment of refractory or
relapsed multiple myeloma was initiated at Duke University Medical Center.
The study is supported by a $2 million grant from the NCI Small Business
Innovation Research Program (SBIR) awarded to Apogee, in conjunction with
Duke University, with additional support from RedHill.

A Phase I/II clinical study evaluating YELIVA(R) in patients with